Applications

1579 – Emicizumab for routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in patients with moderate to severe Haemophilia A (congenital Factor VIII deficiency) WITHOUT Factor VIII inhibitors

Find out about the service or technology in this application and the medical condition it addresses. You can also view the application documents, the deadlines for providing consultation input and the outcome of the application when the MSAC process is complete.

  • Status Complete
  • Type New application
  • Pre-PASC consultation -
  • Pre-MSAC consultation -
  • Outcome Supported

Application details

Reason for application

Health Technology Assessment.

Service or technology in this application

Emicizumab is a monoclonal antibody that is bispecific for Factor IXa and Factor X. The two arms bind to each of these factors, mimicking the action of Factor VIII and allowing the normal clotting cascade to continue. Emicizumab offers the following advantages over the recombinant or plasma-derived factors for patients requiring prophylaxis (Shima 2016):

  • Subcutaneous vs intravenous administration;
  • Weekly vs every other day administration;
  • Sustained plasma concentrations;
  • No risk of FVIII inhibitor development;
  • Activity irrespective of the presence of FVIII inhibitors.

Emicizumab is registered as a prescription medicine by the Therapeutic Goods Administration (TGA).

Type: Therapeutic

Medical condition this application addresses

Haemophilia A (HMA) is an X-linked congenital bleeding disorder caused by a deficiency of the coagulation factor VIII (FVIII). HMA can be mild, moderate or severe depending on the level of deficiency.

  • Mild: usually bleed as a result of injury or major surgery;
  • Moderate: bleed spontaneously but usually as a result of injury;
  • Severe: frequent spontaneous bleeding into muscles and joints.

The mainstay of treatment for HMA is replacement of the deficient Factor VIII by intravenous (IV) recombinant or plasma-derived Factor VIII, either on demand (to treat a bleed) or as prophylaxis. Prophylaxis prevents bleeding and irreversible joint destruction and is considered advisable before HMA patients engage in activities with higher risk of injury. Development of inhibitors (antibodies that neutralise replacement FVIII) is considered the most severe treatment-related complication in HMA, with a lifetime risk of development of ~20–30% in severe HMA and 5–10% in mild or moderate disease (ACHDO 2016).

Application documents

Public summary document

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Meetings to consider this application

  • PASC meeting: 7 to 8 December 2017 
    The population in application 1579 (patients WITHOUT factor VIII inhibitors) was considered as part of PASC’s consideration of application 1510.
  • ESC meeting: 13 to 14 June 2019
  • MSAC meeting: 1 to 2 August 2019